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People with hemophilia B could find their bleeding risk dramatically reduced with just one injection of an experimental gene therapy, a new study reports.

Hemophilia B is a rare and inherited genetic disorder in which people have low levels of the

  • By Dennis Thompson HealthDay Reporter
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  • July 22, 2022
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  • Full Page
  • Gene therapy shows promise in reducing, and even halting, potentially life-threatening bleeding events in people with hemophilia, researchers report.

    Hemophilia A is the most common inherited bleeding disorder, affecting one in 5,000 males worldwide. It's caused by a missing coagulation factor called FVIII.

    The current standard of care involves regular infusions of the FVIII protein...

    Researchers may have found a way for people with severe hemophilia to take their standard treatment less often, if the results of an early trial pan out.

    In what experts called a feat of bioengineering, scientists were able to create a "fusion protein" that may extend the interval between treatments for hemophilia -- from about every couple of days to once a week.

    The early ...

    A new gene therapy appears to serve as a functional cure for the most common type of hemophilia, early clinical trial results indicate.

    Patients who received the one-time intravenous therapy continue to have a more than 90% decrease in bleeding events two to three years after their initial treatment, researchers reported Jan. 1 in the New England Journal of Medicine.

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